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| Name | Class |
|---|---|
| Seelos Therapeutics, Inc. | INDUSTRY |
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The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.
Regimen E will evaluate the safety and efficacy of a single study drug, SLS-005 (Trehalose injection, 90.5 mg/mL for intravenous infusion) in participants with ALS.
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. This trial is designed as a perpetual platform trial. This means that there is a single Master Protocol dictating the conduct of the trial. The HEALEY ALS Platform Trial Master Protocol is registered as NCT04297683.
Once a participant enrolls into the Master Protocol and meets all eligibility criteria, the participant will be eligible to be randomized into any currently enrolling regimen. All participants will have an equal chance of being randomized to any currently enrolling regimen.
If a participant is randomized to Regimen E SLS-005 - Trehalose, the participant will complete a screening visit to assess additional Regimen E eligibility criteria. Once Regimen E eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in a 3:1 ratio to either active SLS-005 or matching placebo.
Regimen E will enroll by invitation, as participants may not choose to enroll in Regimen E. Participants must first enroll into the Master Protocol and be eligible to participate in the Master Protocol before being able to be randomly assigned to Regimen E.
For a list of enrolling sites, please see the HEALEY ALS Platform Trial Master Protocol under NCT04297683.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SLS-005 | Experimental | SLS-005 is administered via infusion once weekly for 24 weeks. |
|
| Matching Placebo | Placebo Comparator | Matching placebo is administered via infusion once weekly for 24 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SLS-005 | Drug | Administration: Infusion Dose: 0.75 g/kg weekly |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Disease Progression as Assessed by the ALSFRS-R Slope | Change in disease severity over time as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function. | 24 Weeks |
| Mortality Event Rate | Mortality is defined as death or death equivalent. A participant is determined to meet the criteria of death equivalent if permanent assisted ventilation (PAV) is used for more than 22 hours per day for more than seven days in a row. The rate of mortality was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times. | Baseline to 24 Weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Respiratory Function | Change in respiratory function over time as measured by Slow Vital Capacity (SVC). | Baseline to 24 Weeks |
| Muscle Strength | Change in muscle strength over time as measured isometrically using hand-held dynamometry (HHD). |
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Inclusion Criteria:
Exclusion Criteria:
The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).
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| Name | Affiliation | Role |
|---|---|---|
| Merit Cudkowicz, MD | Massachusetts General Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Healey Center for ALS at Mass General | Boston | Massachusetts | 02114 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40409314 | Derived | HEALEY ALS Platform Trial; HEALEY ALS Platform Trial Study Group. Safety and efficacy of trehalose in amyotrophic lateral sclerosis (HEALEY ALS Platform Trial): an adaptive, phase 2/3, double-blind, randomised, placebo-controlled trial. Lancet Neurol. 2025 Jun;24(6):500-511. doi: 10.1016/S1474-4422(25)00173-5. |
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| ID | Title | Description |
|---|---|---|
| FG000 | SLS-005 | SLS-005 is administered via infusion once weekly for 24 weeks. SLS-005: Administration: Infusion Dose: 0.75 g/kg weekly |
| FG001 | Matching Placebo | Matching placebo is administered via infusion once weekly for 24 weeks. Matching Placebo: Administration: Infusion Dose: equivalent weight-based volume as described for trehalose |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Baseline Analysis Population includes only placebo participants from the Regimen E Efficacy Regimen Only (ERO) sample; shared placebo participants from other regimens are not included in the Baseline Analysis Population.
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| ID | Title | Description |
|---|---|---|
| BG000 | SLS-005 | SLS-005 is administered via infusion once weekly for 24 weeks. SLS-005: Administration: Infusion Dose: 0.75 g/kg weekly |
| BG001 | Matching Placebo | Matching placebo is administered via infusion once weekly for 24 weeks. Matching Placebo: Administration: Infusion Dose: equivalent weight-based volume as described for trehalose |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Disease Progression as Assessed by the ALSFRS-R Slope | Change in disease severity over time as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function. | Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A (NCT04436497), Regimen B (NCT04436510), Regimen C (NCT04414345) and Regimen D (NCT04615923) contributed placebo participants into the shared placebo cohort used for analysis. | Posted | Mean | Standard Deviation | points per month | 24 Weeks |
|
Up to 35 weeks after participant signed Master Protocol consent.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | SLS-005 | SLS-005 is administered via infusion once weekly for 24 weeks. SLS-005: Administration: Infusion Dose: 0.75 g/kg weekly |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Muscular weakness | Nervous system disorders | MedDRA 23.0 | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Healey Center for ALS Project Management | Healey Center for ALS at Massachusetts General Hospital | 833-425-8257 | (HALT ALS) | healeyalsplatform@mgh.harvard.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Dec 3, 2021 | Jul 11, 2024 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 10, 2023 | Jul 11, 2024 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
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| Matching Placebo |
| Drug |
Administration: Infusion Dose: equivalent weight-based volume as described for trehalose |
|
| Baseline to 24 Weeks |
| Number of Participants That Experienced Death or Death Equivalent | The number of participants who died or met the criterion for a death equivalent from the date of their baseline visit to the end of the Week 24 visit window (generally 175 days after baseline). The death equivalent criterion is the use of permanent assisted ventilation (PAV) for more than 22 hours per day for more than 7 days in a row. | Baseline to 24 Weeks |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| ALS Diagnosis from R El Escorial Criteria | The El Escorial Criteria are a set of guidelines used to diagnose ALS based on clinical signs of upper motor neuron and lower motor neuron dysfunction. | Count of Participants | Participants |
|
| ALS Onset Location | Count of Participants | Participants |
|
| Time Since Symptom Onset at Baseline | Mean | Standard Deviation | Months |
|
| Delay in ALS Symptom Onset and Diagnosis | Mean | Standard Deviation | Months |
|
| Baseline Edaravone Use | Count of Participants | Participants |
|
| Baseline Riluzole Use | Count of Participants | Participants |
|
| ALSFRS-R Total Score | The ALS Functional Rating Score-Revised (ALSFRS-R) measures 12 aspects of physical function. Each of the 12 questions assessing distinct functional ability is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function. | Mean | Standard Deviation | Points |
|
| Baseline Decline in ALSFRS-R | The ALS Functional Rating Score-Revised (ALSFRS-R) measures 12 aspects of physical function. Each of the 12 questions assessing distinct functional ability is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function. Baseline is divided by the number of months between onset of weakness due to ALS and the date of Baseline. A higher number indicates greater decline. | Mean | Standard Deviation | points per month |
|
| SVC | Slow vital capacity is an assessment of breathing function. The total amount of air that can be exhaled is measured. A higher slow vital capacity indicates better breathing function. | Number analyzed in row differs from overall number due to missing data. | Mean | Standard Deviation | percent predicted |
|
| King Stage | The King's ALS Clinical Staging System is a 4-level ordinal scale with the first three levels indicating the number (1, 2, or 3) of distinct central nervous system regions (bulbar, upper limb, and lower limb) with neuromuscular dysfunction. Level 4a indicates nutritional failure and level 4b indicates respiratory failure. Higher scores indicate a worse disease state. | Count of Participants | Participants |
|
| Weight | Mean | Standard Deviation | kg |
|
| Body Mass Index | Mean | Standard Deviation | kg/cm^2 |
|
| Serum Creatinine Concentration | Mean | Standard Deviation | mg/dL |
|
| Serum NfL Concentration | Number analyzed in row differs from overall number due to missing data. | Mean | Standard Deviation | ng/L |
|
| OG001 | Matching Placebo | Matching placebo is administered via infusion once weekly for 24 weeks. Matching Placebo: Administration: Infusion Dose: equivalent weight-based volume as described for trehalose |
|
|
|
| Primary | Mortality Event Rate | Mortality is defined as death or death equivalent. A participant is determined to meet the criteria of death equivalent if permanent assisted ventilation (PAV) is used for more than 22 hours per day for more than seven days in a row. The rate of mortality was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times. | Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A (NCT04436497), Regimen B (NCT04436510), Regimen C (NCT04414345) and Regimen D (NCT04615923) contributed placebo participants into the shared placebo cohort used for analysis. | Posted | Mean | Standard Deviation | events per month | Baseline to 24 Weeks |
|
|
|
| Secondary | Respiratory Function | Change in respiratory function over time as measured by Slow Vital Capacity (SVC). | Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A (NCT04436497), Regimen B (NCT04436510), Regimen C (NCT04414345) and Regimen D (NCT04615923) contributed placebo participants into the shared placebo cohort used for analysis.One SLS-005 participant and 4 placebo participants from FAS were not analyzed due to missing data. | Posted | Least Squares Mean | Standard Error | percent change | Baseline to 24 Weeks |
|
|
|
|
| Secondary | Muscle Strength | Change in muscle strength over time as measured isometrically using hand-held dynamometry (HHD). | Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A (NCT04436497), Regimen B (NCT04436510), Regimen C (NCT04414345) and Regimen D (NCT04615923) contributed placebo participants into the shared placebo cohort used for analysis. Two placebo participants from FAS were not analyzed due to missing data. | Posted | Least Squares Mean | Standard Error | percent change | Baseline to 24 Weeks |
|
|
|
|
| Secondary | Number of Participants That Experienced Death or Death Equivalent | The number of participants who died or met the criterion for a death equivalent from the date of their baseline visit to the end of the Week 24 visit window (generally 175 days after baseline). The death equivalent criterion is the use of permanent assisted ventilation (PAV) for more than 22 hours per day for more than 7 days in a row. | Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A (NCT04436497), Regimen B (NCT04436510), Regimen C (NCT04414345) and Regimen D (NCT04615923) contributed placebo participants into the shared placebo cohort used for analysis. | Posted | Count of Participants | Participants | Baseline to 24 Weeks |
|
|
|
|
| 6 |
| 120 |
| 19 |
| 120 |
| 107 |
| 120 |
| EG001 | Matching Placebo | Matching placebo is administered via infusion once weekly for 24 weeks. Matching Placebo: Administration: Infusion Dose: equivalent weight-based volume as described for trehalose | 0 | 41 | 3 | 41 | 33 | 41 |
| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Amyotrophic lateral sclerosis | Nervous system disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Cerebrovascular accident | Nervous system disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Syncope | Nervous system disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| COVID-19 | Infections and infestations | MedDRA 23.0 | Non-systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 23.0 | Non-systematic Assessment |
|
| Hip fracture | Injury, poisoning and procedural complications | MedDRA 23.0 | Non-systematic Assessment |
|
| Subdural haemorrhage | Injury, poisoning and procedural complications | MedDRA 23.0 | Non-systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Colitis ischaemic | Gastrointestinal disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Complication associated with device | General disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Urticaria | Immune system disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Breast cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 23.0 | Non-systematic Assessment |
|
| Hypertension | Vascular disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Neuromyopathy | Nervous system disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Dysarthria | Nervous system disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA 23.0 | Non-systematic Assessment |
|
| Infusion site bruising | Injury, poisoning and procedural complications | MedDRA 23.0 | Non-systematic Assessment |
|
| Post lumbar puncture syndrome | Injury, poisoning and procedural complications | MedDRA 23.0 | Non-systematic Assessment |
|
| Fatigue | General disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Infusion related reaction | General disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Oedema peripheral | General disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Dysphagia | Gastrointestinal disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Salivary hypersecretion | Gastrointestinal disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Abdominal discomfort | Gastrointestinal disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| COVID-19 | Infections and infestations | MedDRA 23.0 | Non-systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA 23.0 | Non-systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Non-systematic Assessment |
|
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Non-systematic Assessment |
|
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| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Clinically Probable ALS |
|
| Clinically Probable ALS - Laboratory Supported |
|
| Multiple |
|
| Respiratory |
|
| 3 Region with Neuromuscular Dysfunction |
|
| 4a/b Nutritional/Respiratory Failure |
|